{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,20]],"date-time":"2026-05-20T17:30:48Z","timestamp":1779298248871,"version":"3.51.4"},"reference-count":144,"publisher":"MDPI AG","issue":"7","license":[{"start":{"date-parts":[[2024,6,27]],"date-time":"2024-06-27T00:00:00Z","timestamp":1719446400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Medicina"],"abstract":"<jats:p>Muscular dystrophies (MDs) are a heterogeneous group of diseases of genetic origin characterized by progressive skeletal muscle degeneration and weakness. There are several types of MDs, varying in terms of age of onset, severity, and pattern of the affected muscles. However, all of them worsen over time, and many patients will eventually lose their ability to walk. In addition to skeletal muscle effects, patients with MDs may present cardiac and respiratory disorders, generating complications that could lead to death. Interdisciplinary management is required to improve the surveillance and quality of life of patients with an MD. At present, pharmacological therapy is only available for Duchene muscular dystrophy (DMD)\u2014the most common type of MD\u2014and is mainly based on the use of corticosteroids. Other MDs caused by alterations in dystrophin-associated proteins (DAPs) are less frequent but represent an important group within these diseases. Pharmacological alternatives with clinical potential in patients with MDs and other proteins associated with dystrophin have been scarcely explored. This review focuses on drugs and molecules that have shown beneficial effects, mainly in experimental models involving alterations in DAPs. The mechanisms associated with the effects leading to promising results regarding the recovery or maintenance of muscle strength and reduction in fibrosis in the less-common MDs (i.e., with respect to DMD) are explored, and other therapeutic targets that could contribute to maintaining the homeostasis of muscle fibers, involving different pathways, such as calcium regulation, hypertrophy, and maintenance of satellite cell function, are also examined. It is possible that some of the drugs explored here could be used to affordably improve the muscular function of patients until a definitive treatment for MDs is developed.<\/jats:p>","DOI":"10.3390\/medicina60071060","type":"journal-article","created":{"date-parts":[[2024,6,27]],"date-time":"2024-06-27T11:19:02Z","timestamp":1719487142000},"page":"1060","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":3,"title":["Pharmacological Treatments and Therapeutic Targets in Muscle Dystrophies Generated by Alterations in Dystrophin-Associated Proteins"],"prefix":"10.3390","volume":"60","author":[{"given":"Alexandra","family":"Luna-Angulo","sequence":"first","affiliation":[{"name":"Divisi\u00f3n de Neurociencias Clinicas, Instituto Nacional de Rehabilitaci\u00f3n \u201cLuis Guillermo Ibarra Ibarra\u201d, Calzada M\u00e9xico-Xochimilco, No. 289, Arenal de Guadalupe, Tlalpan, Ciudad de M\u00e9xico 14389, Mexico"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Carlos","family":"Landa-Sol\u00eds","sequence":"additional","affiliation":[{"name":"Unidad de Ingenier\u00eda de Tejidos, Terapia Celular y Medicina Regenerativa, Divisi\u00f3n de Biotecnolog\u00eda, Instituto Nacional de Rehabilitaci\u00f3n \u201cLuis Guillermo Ibarra Ibarra\u201d, Calzada M\u00e9xico-Xochimilco, No. 289, Arenal de Guadalupe, Tlalpan, Ciudad de M\u00e9xico 14389, Mexico"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Rosa Elena","family":"Escobar-Cedillo","sequence":"additional","affiliation":[{"name":"Departamento de Electromiograf\u00eda y Distrofia Muscular, Instituto Nacional de Rehabilitaci\u00f3n \u201cLuis Guillermo Ibarra Ibarra\u201d, Calzada M\u00e9xico-Xochimilco, No. 289, Arenal de Guadalupe, Tlalpan, Ciudad de M\u00e9xico 14389, Mexico"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Francisco Javier","family":"Estrada-Mena","sequence":"additional","affiliation":[{"name":"Laboratorio de Biolog\u00eda Molecular, Universidad Panamericana, Facultad de Ciencias de la Salud, Augusto Rodin 498, Ciudad de M\u00e9xico 03920, Mexico"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4268-2279","authenticated-orcid":false,"given":"Laura","family":"S\u00e1nchez-Chapul","sequence":"additional","affiliation":[{"name":"Divisi\u00f3n de Neurociencias Clinicas, Instituto Nacional de Rehabilitaci\u00f3n \u201cLuis Guillermo Ibarra Ibarra\u201d, Calzada M\u00e9xico-Xochimilco, No. 289, Arenal de Guadalupe, Tlalpan, Ciudad de M\u00e9xico 14389, Mexico"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2509-4923","authenticated-orcid":false,"given":"Benjam\u00edn","family":"G\u00f3mez-D\u00edaz","sequence":"additional","affiliation":[{"name":"Departamento de Medicina Gen\u00f3mica, Instituto Nacional de Rehabilitaci\u00f3n \u201cLuis Guillermo Ibarra Ibarra\u201d, Calzada M\u00e9xico-Xochimilco, No. 289, Arenal de Guadalupe, Tlalpan, Ciudad de M\u00e9xico 14389, Mexico"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Paul","family":"Carrillo-Mora","sequence":"additional","affiliation":[{"name":"Divisi\u00f3n de Neurociencias Clinicas, Instituto Nacional de Rehabilitaci\u00f3n \u201cLuis Guillermo Ibarra Ibarra\u201d, Calzada M\u00e9xico-Xochimilco, No. 289, Arenal de Guadalupe, Tlalpan, Ciudad de M\u00e9xico 14389, Mexico"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hamlet","family":"Avil\u00e9s-Arnaut","sequence":"additional","affiliation":[{"name":"Facultad de Ciencias Biol\u00f3gicas de la Universidad Aut\u00f3noma de Nuevo Leon, Av. Universidad s\/n Ciudad Universitaria, San Nicolas de los Garza 66455, Mexico"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Livier","family":"Jim\u00e9nez-Hern\u00e1ndez","sequence":"additional","affiliation":[{"name":"LJ Comunicaci\u00f3n Gr\u00e1fica, Agua Marina #2827, Residencial Victoria, Guadalajara 44986, Mexico"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Dulce Ade\u00ed","family":"Jim\u00e9nez-Hern\u00e1ndez","sequence":"additional","affiliation":[{"name":"LJ Comunicaci\u00f3n Gr\u00e1fica, Agua Marina #2827, Residencial Victoria, Guadalajara 44986, Mexico"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6530-4636","authenticated-orcid":false,"given":"Antonio","family":"Miranda-Duarte","sequence":"additional","affiliation":[{"name":"Departamento de Medicina Gen\u00f3mica, Instituto Nacional de Rehabilitaci\u00f3n \u201cLuis Guillermo Ibarra Ibarra\u201d, Calzada M\u00e9xico-Xochimilco, No. 289, Arenal de Guadalupe, Tlalpan, Ciudad de M\u00e9xico 14389, Mexico"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1968","published-online":{"date-parts":[[2024,6,27]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"2025","DOI":"10.1016\/S0140-6736(19)32910-1","article-title":"Muscular dystrophies","volume":"394","author":"Mercuri","year":"2019","journal-title":"Lancet"},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"687","DOI":"10.1016\/S0140-6736(02)07815-7","article-title":"The muscular dystrophies","volume":"359","author":"Emery","year":"2002","journal-title":"Lancet"},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"96","DOI":"10.1186\/s13018-022-02996-8","article-title":"Global prevalence of Duchenne and Becker muscular dystrophy: A systematic review and meta-analysis","volume":"17","author":"Salari","year":"2022","journal-title":"J. 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